To those who say “impossible, impractical, unrealistic,” we say:
We are developing a new class of innovative medicines called
We’re committed to
ensuring broad patient
We are focused on diseases for which treatment options are limited or simply don't exist.
Updated as of October 2021
Our robust pipeline contains therapies in late-stage (Phase 3) and early-stage (Phase 2 or earlier) trials. Our RNAi therapies leverage two distinct approaches to enable delivery of small interfering Ribonucleic Acid (siRNA): lipid nanoparticles (LNP) and GalNAc-conjugates.